Neurofibromatosis Type 1Phase 4NCT00169611
NF1-Attention: Study of Children With Neurofibromatosis Type 1 Treated by Methylphenidate
A Phase 4 study testing methylphenidate in people with Neurofibromatosis Type 1, run by Hospices Civils de Lyon at 1 site in France.
Trial summary
Neurofibromatosis type 1 (NF1) is an autosomal dominant disorder with an estimated prevalence of 1/2190 to 1/6711. Attention deficit hyperactivity disorder (ADHD) has been reported to be common in NF1. Read more about this study
- Who is it for?
- Ages 7 to 12 years
- Conditions the study is for
- Neurofibromatosis Type 1
- What is being tested?
- methylphenidate
- How many people
- 80 participants
- Where
- 1 siteFrance
- Study dates
- Jan 2004to Jan 2011
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 7 to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age 7-12 years
- IQ between 80-120.
- Gender: male or female
- Children with neurofibromatosis type 1 (according to the National Institutes of Health [NIH] 1988).
- Patients with school difficulties pointed out by parents or teachers
- Patients with attention difficulties as defined by anamnesis
Who cannot join
- IQ > 120 or IQ < 80
- Child depression
- Unwillingness to participate
- Patients with cerebral complication of neurofibromatosis type 1 (chiasma glioma, moya-moya) as detected by cerebral magnetic resonance imaging (MRI).
- Participation in another study
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Comportemental and Neuropsychologic Study of Children With Neurofibromatosis Type 1 Treated by Methylphenidate. A Double-blind Randomised Study Methylphenidate Versus Placebo
Neurofibromatosis type 1 (NF1) is an autosomal dominant disorder with an estimated prevalence of 1/2190 to 1/6711. Attention deficit hyperactivity disorder (ADHD) has been reported to be common in NF1. We, the researchers at Hospices Civils de Lyon, designed a randomized, double blind, placebo controlled, crossover trial with a total follow-up duration of 9 weeks to evaluate the effect of methylphenidate (MPH) on the improvement on the simplified parents Conners' Rating Scale. In a parallel exploratory study we will compare the nature of attention deficit disorders in NF1 children to 30 ADHD NF1-free controls. Children aged 7 to 12 years are eligible when their intelligence quotient (IQ) is between 80 and 120. Fifty subjects (25 for each period) were required for testing the primary study hypothesis.
- Conditions
- Neurofibromatosis Type 1
- Being tested
- methylphenidate
- Keywords
- Neurofibromatosis type 1, Attention deficit with hyperactivity disorder school difficulties, methylphenidate
- Medical subject headings
- Neurofibromatosis 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
methylphenidateDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
Effect of methylphenidate compared to placebo on ADHD. Time of measurements are realised on day 0, day 28, day 63 + 2 days.
Secondary outcomes (3)
To evaluate the efficacy of methylphenidate compared to placebo on specific neuropsychologic, depression and anxiety scales
To compare the nature of attention deficit disorders in NF1 children with those with primary ADHD, depression, and anxiety using the State Trait Anxiety Inventory for Children (STAI-C) and the Children's Depression Inventory (CDI).
Time of measurements are realised on day 0, day 28, day 63 + 2 days.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Sep 2005 (estimated)
- Study startJan 2004
- Primary completionJan 2011
- Study completionJan 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Neurofibromatosis Type 1Studies of the same condition, open and under way.
- More trials run by Hospices Civils de LyonThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hospices Civils de Lyon
- Sponsor type
- University, hospital or other
- Investigators
- Laurence LION-FRANCOIS, MD, Principal investigator, Service de Neuropédiatrie - Hôpital Femme Mère Enfant - Hospices Civils de Lyon - 59 bd Pinel - 69677 BRON - FranceIsabelle KEMLIN, Principal investigator, Service de Neuropédiatrie - Hôpital Armand Trousseau - PARIS - France
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00169611
- Sponsor's protocol ID
- 2003.310
- Status last verified
- Jan 2011
- First submitted
- 9 Sep 2005
- Last updated
- 27 Jan 2011
- Results posted
- No
Publications
Lion-Francois L, Gueyffier F, Mercier C, Gerard D, Herbillon V, Kemlin I, Rodriguez D, Ginhoux T, Peyric E, Coutinho V, Breant V, des Portes V, Pinson S, Combemale P, Kassai B; Reseau NF1 Rhone Alpes Auvergne-France. The effect of methylphenidate on neurofibromatosis type 1: a randomised, double-blind, placebo-controlled, crossover trial. Orphanet J Rare Dis. 2014 Sep 10;9:142. doi: 10.1186/s13023-014-0142-4.
PubMed 25205361
Study at a glance
From the registry record, last updated by the sponsor on 27 Jan 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Comportemental and Neuropsychologic Study of Children With Neurofibromatosis Type 1 Treated by Methylphenidate. A Double-blind Randomised Study Methylphenidate Versus Placebo
- ClinicalTrials.gov ID
- NCT00169611
- Acronym / sponsor ID
- 2003.310
- Conditions
- Neurofibromatosis Type 1
- Keywords
- Neurofibromatosis type 1, Attention deficit with hyperactivity disorder school difficulties, methylphenidate
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- Hospices Civils de LyonUniversity, hospital or other
- Study start
- Jan 2004
- Primary completion
- Jan 2011 (actual)
- Study completion
- Jan 2011 (actual)
- First posted
- 15 Sep 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Neurofibromatosis type 1 (NF1) is an autosomal dominant disorder with an estimated prevalence of 1/2190 to 1/6711. Attention deficit hyperactivity disorder (ADHD) has been reported to be common in NF1. We, the researchers at Hospices Civils de Lyon, designed a randomized, double blind, placebo controlled, crossover trial with a total follow-up duration of 9 weeks to evaluate the effect of methylphenidate (MPH) on the improvement on the simplified parents Conners' Rating Scale. In a parallel exploratory study we will compare the nature of attention deficit disorders in NF1 children to 30 ADHD NF1-free controls. Children aged 7 to 12 years are eligible when their intelligence quotient (IQ) is between 80 and 120. Fifty subjects (25 for each period) were required for testing the primary study hypothesis.
Study design
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
Who may qualify
- Ages
- 7 to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Age 7-12 years
- IQ between 80-120.
- Gender: male or female
- Children with neurofibromatosis type 1 (according to the National Institutes of Health [NIH] 1988).
- Patients with school difficulties pointed out by parents or teachers
Key exclusion criteria (5 listed)
- IQ > 120 or IQ < 80
- Child depression
- Unwillingness to participate
- Patients with cerebral complication of neurofibromatosis type 1 (chiasma glioma, moya-moya) as detected by cerebral magnetic resonance imaging (MRI).
- Participation in another study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| methylphenidate | Drug | – |
Primary outcome
Effect of methylphenidate compared to placebo on ADHD. Time of measurements are realised on day 0, day 28, day 63 + 2 days.
3 secondary outcomes
To evaluate the efficacy of methylphenidate compared to placebo on specific neuropsychologic, depression and anxiety scales
To compare the nature of attention deficit disorders in NF1 children with those with primary ADHD, depression, and anxiety using the State Trait Anxiety Inventory for Children (STAI-C) and the Children's Depression Inventory (CDI).
Time of measurements are realised on day 0, day 28, day 63 + 2 days.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jan 2011.
- Derived from this study: PubMed 25205361Lion-Francois L, Gueyffier F, Mercier C, Gerard D, Herbillon V, Kemlin I, Rodriguez D, Ginhoux T, Peyric E, Coutinho V, Breant V, des Portes V, Pinson S, Combemale P, Kassai B; Reseau NF1 Rhone Alpes Auvergne-France. The effect of methylphenidate on neurofibromatosis type 1: a randomised, double-blind, placebo-controlled, crossover trial. Orphanet J Rare Dis. 2014 Sep 10;9:142. doi: 10.1186/s13023-014-0142-4.
- Related InfoLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Laurence LION-FRANCOIS, MD principal investigator
Service de Neuropédiatrie - Hôpital Femme Mère Enfant - Hospices Civils de Lyon - 59 bd Pinel - 69677 BRON - France
Isabelle KEMLIN principal investigator
Service de Neuropédiatrie - Hôpital Armand Trousseau - PARIS - France
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing methylphenidate in people with Neurofibromatosis Type 1, run by Hospices Civils de Lyon at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Jan 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Effect of methylphenidate compared to placebo on ADHD. Time of measurements are realised on day 0, day 28, day 63 + 2 days.. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00169611, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Jan 2011.